FDA Approves Duvyzat (givinostat) for Duchenne Treatment

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The U.S. Food and Drug Administration (FDA) approved Duvyzat (givinostat) for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat individuals with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor developed by Italfarmaco, and works by targeting pathogenic processes to reduce inflammation and loss of muscle. 

Approval for Duvystat was based on a randomized, double-blind, placebo-controlled Phase 3 study called EPIDYS. That study, which was completed in 2022, met the primary endpoint in change from baseline to month 18 using a four-stair climb to measure muscle function. Ambulant individuals with Duchenne (aged 6 and older) taking givinostat demonstrated a slower decline in performing this task over 18 months compared to those receiving placebo.  

Secondary endpoints that evaluated muscle function and strength (e.g. NSAA; time to rise; and fat infiltration) also showed favorable results in the givinostat-treated cohort compared to the control group. There were minimal safety concerns. 

The results of the EPIDYS study were recently published in The Lancet Neurology.

In anticipation of this approval, Italfarmaco S.p.A (headquartered in Milan, Italy) launched a new division in the United States called ITF Therapeutics, LLC, which will focus on US patient access to Duvyzat.  ITF Therapeutic’s website should be live soon.  

Duvyzat is the eighth FDA-approved therapy for Duchenne following the approval of AGAMREE (vamorolone) in October 2023. CureDuchenne is delighted to see the approval of another drug that can slow down the progression of Duchenne and look forward to seeing continual progress on the road towards a cure.  

See the Italfarmaco press release below.


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